Researchers Suggest A Way to Test the Safety of Stem Cell Therapy in Progressive Supranuclear Palsy

A recent publication in the Journal of Translational Medicine has highlighted the potential for stem cells to help patients with progressive supranuclear palsy and has proposed measures for testing the safety of stem cell therapy. Like Parkinson’s disease, progressive supranuclear palsy is a neurodegenerative disease that is progressive and characterized by abnormal deposits of the protein tau in the brain. Also like Parkinson’s, there is not a reliable prevention or cure for the disease.

Previous studies have shown that stem cells can help improve Parkinson’s disease symptoms, including problematic functioning of the brain chemical dopamine. Though the etiology of progressive supranuclear palsy is unknown, because it has some physiological features that are similar to Parkinson’s, researchers have reasoned that stem cell therapies may also help the patients who suffer from this disease. However, given that Parkinson’s disease and progressive supranuclear palsy can be differentiated on a clinical basis, it was initially unclear what specific impact stem cells may have on progressive supranuclear palsy.

Nonetheless, consistent with the idea that stem cells may help those with progressive supranuclear palsy, some studies have provided promising data for the ability of stem cells to improve relevant symptoms. The aim of the recent Journal of Translational Medicine publication was to lay out a plan for testing the safety of stem cell therapy in progressive supranuclear palsy patients.

The researchers propose a randomized, double-blind phase I clinical trial that would also be placebo-controlled to assess the safety of stem cell therapy using mesenchymal stem cells in patients with progressive supranuclear palsy patients. In addition to providing evidence on the safety of the stem cell therapy, another value in executing the proposal from this publication is the ability to distinguish whether the effects of stem cells in progressive supranuclear palsy that have been previously observed are due to placebo. In other words, this placebo-controlled trial would allow researchers to determine whether patients improve because they believe they are going to improve or because the stem cell therapy has a direct impact on their disease.